Registries for European Reference Networks (ERNs) Hélène Le Borgne, Policy officer, ERN Team, DG SANTE, European Commission
Why the European Reference Networks (ERNs)? • Many of those affected by a rare or complex condition do not have access to diagnosis and high-quality treatment • Expertise and specialist knowledge may be scarce as patient numbers are low • Important delay in diagnosis because of lack of care pathways, diagnostic capacity (no tests available) and treatments in many cases • No country alone has the knowledge and capacity to treat all rare and low prevalence complex diseases "The knowledge travels, not the patient ” Classified as internal/staff & contractors by the European Medicines Agency
Launch of the first ERNs in 2017 Legal basis: Directive on patients’ rights in cross-border healthcare Chapter IV - Cooperation between MS Article 12 - European Reference Networks Application and evaluation process from March 2016 to March 2017: 24 ERNs 26 Countries Classified as internal/staff & contractors by the European Medicines Agency
https://ec.europa.eu/health/ern_en ERN BOND Bone Diseases ERN EuroBloodNet Onco-Hematological Diseases ERN CRANIO Craniofacial anomalies and ENT ERN EUROGEN Urogenital Diseases disorders ERN EURO-NMD Neuromuscular Diseases Endo-ERN Endocrine Conditions ERN GUARD-HEART Diseases of the Heart ERN EpiCARE Rare and Complex Epilepsies ERN ITHACA Congenital Malformations and Intellectual ERKNet Kidney Diseases Disability ERN GENTURIS Genetic Tumour Risk Syndromes MetabERN Hereditary metabolic diseases ERN-EYE Eye Diseases ERN PaedCan Paediatric Cancer ERN RARE-LIVER Hepatological Diseases ERNICA inherited and congenital anomalies ERN ReCONNET Connective Tissue and Musculoskeletal ERN-LUNG Respiratory Diseases Diseases ERN RITA Immunodeficiency, Auto-Inflammatory and Auto ERN-RND Neurological Diseases Immune Diseases ERN-Skin Skin Disorders ERN TRANSPLANT-CHILD Transplantation in Children ERN EURACAN Solid Adult Cancers VASCERN Multisystemic Vascular Diseases Classified as internal/staff & contractors by the European Medicines Agency
ERNs applied for specific (groups of) diseases • ERN EURACAN : rare neoplasms of the: connective tissue (G1), female genital organs and placenta (G2), male genital organs & urinary tract (G3), neuroendocrine system (G4), digestive tract (G5), endocrine organs (G6), head & neck (G7), thorax (G8), skin & eye melanoma (G9), brain, spinal cords (G10) • ERN PaedCan : leukemias, myeloproliferative and myelodysplastic diseases, lymphomas & reticuloendothelial neoplasms, CNS + miscell. intracranial & intraspinal neoplasms, neuroblastoma & other peripheral nervous cell tumors, retinoblastoma, renal tumors, hepatic tumors, malignant bone tumors, soft tissue and other extraosseous sarcomas, germ cell tumors, trophoblastic tumors and neoplasms of gonads, other malignant epithelial neoplasms & malignant melanomas, other unspecified malignant neoplasms (Hematopoietic and Lymphoid: LCH), hepatoblastoma, very rare tumours, nephroblastoma, neuroblastoma, brain tumours, Ewing sarcoma, soft tissue sarcoma, germ cell tumour, Langerhans cell histiocytosis • ERN EuroBloodNet : 1) non-oncological rare hematological diseases: rare blood cell defect, bone marrow failures, bleeding and coagulation disorders, hereditary haemacrhomatosis and iron metabolism related disorders; 2) oncological rare hematological diseases: lymphoid and myeloid malignancies Classified as internal/staff & contractors by the European Medicines Agency
What do the ERNs do? ✓ Exchange of expertise and clinical data on patient cases ✓ Virtual remote consultations through the IT tool developed by the EC: Clinical Patient Management System (CPMS) ✓ Research → registries ! ✓ Clinical guidelines ✓ Education & training Classified as internal/staff & contractors by the European Medicines Agency
Added value of the ERNs Give patients and doctors across the EU access to the best expertise and timely exchange of knowledge, without having to travel to another country. ✓ increase the likelihood of early and accurate diagnosis and effective treatment ✓ improve public and professional awareness of rare and complex diseases ✓ platforms for the development of guidelines, training and knowledge-sharing ✓ facilitate large clinical studies to improve understanding and develop new drugs ✓ opportunity for networking with likeminded experts from across Europe — ending professional isolation Classified as internal/staff & contractors by the European Medicines Agency •
Governance of the ERN Initiative (1) European Commission ✓ Co-definition of the legal framework for the establishment of the Networks / implementation of the procedures ✓ Provision of support to the governance bodies ✓ Funding of the initiative (the coordination of the Networks – the IT tools, other support activities …) ERN Board of Member States ✓ Endorse the healthcare providers wishing to become members of the ERNs ✓ Approve the Networks and their membership ✓ Steer the initiative Classified as internal/staff & contractors by the European Medicines Agency
Governance of the ERN Initiative (2) European Reference Networks (ERNs) ✓ Coordinators (ERN Coordinators ’ Group) ✓ Members - in each ERN: A Board, Task Forces / Working Groups etc. ✓ Patients ’ representatives ✓ Hospital managers Working groups, composed of representatives of the BoMS and of the ERN Coordinators WG on Integration (in national healthcare systems) WG on Research + established in 2019: Registries' Task Force (close cooperation with JRC Rare Disease Registration Platform) WG on Legal & Ethical issues, relations with Stakeholders WG on Knowledge generation (education and training) WG on Monitoring Classified as internal/staff & contractors by the European Medicines Agency
Registries for ERNs Five ERNs already funded – started in 2018 ✓ ERKNet: ERKReg project - ERKNet Registry for Rare Kidney Diseases ✓ MetabERN: U-IMD project - Unified European Registry for Inherited Metabolic Disorders ✓ Endo-ERN: EuRRECa project - European Registries for rare Endocrine Conditions ✓ ERN-LUNG: RD Registry Data Warehouse project ✓ ERN PaedCan: PARTNER (Paediatric Rare Tumours Network – European Registry) Ongoing call for the 19 other ERNs ✓ Call for grants under the EU Health Programme (10/10/2019) → to start mid 2020 ✓ ERN Registries Task Force created under the ERN Research Working Group ✓ Strong cooperation with EU Rare Disease Platform and tools developed by Joint Research Centre (JRC) of the Commission with DG SANTE ✓ Synergies with EU-funded Research "European Joint Programme for Rare Diseases" (EJP RD) Classified as internal/staff & contractors by the European Medicines Agency
For a common ground for all ERNs: EU Rare Disease Registration Platform and JRC "Common Data Elements" • https://eu-rd-platform.jrc.ec.europa.eu/ Classified as internal/staff & contractors by the European Medicines Agency
For a common ground for all ERNs: JRC "Common Data Elements" (1) • https://eu-rd-platform.jrc.ec.europa.eu/set-of-common-data-elements Classified as internal/staff & contractors by the European Medicines Agency
For a common ground for all ERNs: JRC "Common Data Elements" (2) • https://eu-rd-platform.jrc.ec.europa.eu/set-of-common-data-elements Classified as internal/staff & contractors by the European Medicines Agency
Cancer & rare diseases are 2 'use cases' under the "1 Million Genome Initiative" • https://ec.europa.eu/digital-single-market/en/european-1-million-genomes-initiative Classified as internal/staff & contractors by the European Medicines Agency
Cancer: a priority at EU level ✓ Joint Action on rare cancers (JARC) : Final meeting last Sept. 2019, close collaboration with ERNs ✓ Health Commissioner Vytenis Andriukaitis at ECCO Summit • President-Elect Ursula Von der Leyen: ✓ In her 'political guidelines' announced an 'Action Plan against Cancer' ✓ In the mission letter to Stella Kyriakides, Commissioner-designate for Health: "I want you to put forward Europe’s Beating Cancer Plan to support Member States to improve cancer prevention and care. This should propose actions to strengthen our approach at every key stage of the disease: prevention, diagnosis, treatment, life as a cancer survivor and palliative care. There should be a close link with the research mission on cancer in the future Horizon Europe programme ." Cancer mission ✓ "the European research and innovation missions aim to deliver solutions to some of the greatest challenges facing our world, such as cancer" ✓ Chaired by Nobel Prize Winner Prof. Harald zur Hausen; Prof. Ruth Ladenstein (Coordinator ERN PaedCan) member of the Board Classified as internal/staff & contractors by the European Medicines Agency
• Thank you! Classified as internal/staff & contractors by the European Medicines Agency
Recommend
More recommend
